DESCRIPTION: (Applicant's Description) The goals of the Translational Research Core are to: (1) provide high quality, well-characterized vectors for animal and in vitro experiments for Projects 2 and 3; (2) provide clinical grade vectors (produced under GMP conditions) for Project 1, and (3) conduct toxicology studies under GLP conditions for promising vectors developed. To support the translational process of moving the proposed projects into phase I human clinical trials, a Translational Core has been implemented. This will consist of multiple units that already function with similar goals in the Institute for Human Gene Therapy at the University of Pennsylvania: The Vector Development Core which will assist in the molecular cloning, and selection of multiple vectors and initial scale-up of vectors as outlined in Project 2; a vector production group that will take limited numbers of these vectors and produce the under Good Manufacturing Practices (GMP) as required by the FDA for final pre-clinical safety studies as well as for human clinical trials; and a toxicology group that will conduct the safety assessment studies needed to assess potential toxicological problems that may occur in humans. These three units will perform the final development stage work under the expected regulations expected by the FDA, as well as provide the documentation for IND application in the form of summaries for the IND and detailed Final Reports for toxicology studies and Drug Master Files for selected vectors.